International Journal of Molecular Medical Science, 2024, Vol.14, No.2, 81-89 http://medscipublisher.com/index.php/ijmms 84 One of the most famous clinical trials is aimed at β-mediterranean anemia treatment (Khiabani et al., 2023). Ninety percent of β-thalassemia is caused by point mutations in the β-globin gene, with a minority resulting from larger segment deletions or duplications (Figure 2). In this trial, researchers use the CRISPR-Cas system to edit patients' hematopoietic stem cells to correct the mutated genes within the patients. The aim of this trial is to provide a potential therapeutic approach for patients with β-thalassemia, aiming to alleviate or eliminate their symptoms. Figure 2 Causes β- Gene deletion in thalassemia Additionally, there are several other ongoing clinical trials involving the use of the CRISPR-Cas system to treat other genetic diseases, such as cystic fibrosis, hereditary deafness, and hereditary retinal disorders. These trials aim to assess the safety and efficacy of the CRISPR-Cas system in treating these diseases, providing new therapeutic options for gene therapy. However, it is important to note that there are still challenges and limitations in clinical applications. The precision and efficiency of the CRISPR-Cas system need further improvement to ensure accurate editing of target genes and avoid adverse effects on other genes. Furthermore, safety is a crucial consideration, requiring assurance that the use of the CRISPR-Cas system does not trigger adverse reactions or potential side effects. Additionally, ethical and legal issues need careful consideration to ensure that the application of gene therapy is rational and ethical. 2.2 Clinical applications of genome editing Clinical applications of the CRISPR-Cas system in genome editing have made progress, yet further research and clinical trials are necessary to assess its safety and efficacy. Genome editing involves precise modifications and repairs of the human genome using the CRISPR-Cas system, aiming to treat genetic diseases or enhance human health. In clinical research, the CRISPR-Cas system has been employed for the treatment of certain genetic diseases. For instance, as mentioned earlier, it has been applied in the treatment of β-thalassemia. Additionally, the CRISPR-Cas system has been utilized in the treatment of other genetic disorders. By editing and repairing the patient's genome, researchers aim to address the root causes of these diseases, ultimately achieving therapeutic outcomes. 2.3 Prospects of personalized medicine and precision gene editing The advancements of the CRISPR-Cas system in clinical research have opened significant prospects for personalized medicine and precision gene editing. Personalized medicine involves tailoring medical treatments to individual patients based on their genomic information and other relevant factors. The emergence of the CRISPR-Cas system has enhanced the precision and efficiency of genome editing, providing a robust tool for personalized medicine. With continuous technological development, personalized medicine and precision gene editing are poised to become routine therapeutic approaches, offering improved treatment outcomes and quality of life for patients with various diseases. Through the CRISPR-Cas system, doctors can precisely edit and repair specific gene mutations in patients,
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